Human Genome Sciences and its partner GlaxoSmithKline revealed that the high dose of their experimental lupus therapy demonstrated a statistically superior response compared to placebo in a second late-stage trial, laying the groundwork for an FDA marketing application.
Benlysta has helped transform HGS's prospects, and analysts were quick to note that confirmatory Phase III data may well clear the way to the first new drug approval for lupus in more than 40 years. HGS Chief Executive Officer Tom Watkins (photo) has already laid out plans to charge around $20,000 a year for the therapy, creating a market worth billions of dollars. The two companies plan to ask for regulatory approval in the first half of 2010.
"Benlysta is poised to capture a multibillion-dollar opportunity for lupus," Joseph Schwartz, an analyst for Leerink Swann, noted just days ago. HGS stock (HGSI) soared 40 percent in pre-market trading on the news. But while the end result was positive, the data also wasn't the unqualified success HGS had seen in the first Phase III trial.
HGS tested the drug in a late-stage trial dubbed Bliss-76 in 819 patients. The higher dose group demonstrated a 43.2 percent response rate compared to 33.8 percent in the placebo group. Unlike the first late-stage trial, patients taking the low-dose formulation of the drug did not demonstrate a statistically superior response. One of the patients taking the high dose died, as did two patients taking the low dose. "The BLISS-76 results confirm our view that Benlysta has the potential to become the first new approved drug in decades for people living with systemic lupus," said Watkins.
"The lupus community has waited for decades for one positive phase three trial of an investigative drug developed for lupus," Dr. Joan T. Merrill, a lupus expert at the Oklahoma Medical Research Foundation and an investigator in the trial. "Now we have two."